Ammonul News

Ammonul Q&A

ADVERSE REACTIONS

The safety data were obtained from 316 patients who received AMMONUL® as emergency (rescue) or prospective treatment for hyperammonemia as part of an uncontrolled, open-label study. The study population included patients between the ages of 0 to 53 years with a mean (SD) of 6.2 (8.54) years; 51% were male and 49% were female who had the following diagnoses: OTC (46%), ASS (22%), CPS (12%), ASL (2%), ARG (< 1%), THN (< 1%), and other (18%).

Clinically Important Adverse Reactions

Adverse events occurred most frequently in the following system organ classes: nervous system disorders (22% of patients), metabolism and nutrition disorders (21% of patients), and respiratory, thoracic and mediastinal disorders (15% of patients). The most frequently reported adverse events were vomiting (9% of patients), hyperglycemia (7% of patients), hypokalemia (7% of patients), convulsions (6% of patients), and mental impairment (6% of patients).

Subpopulation and Risk Factor Data

Adverse events were reported with similar frequency in patients with OTC, ASS, CPS, and diagnoses categorized as "other." Nervous system disorders were more frequent in patients with OTC and CPS, compared with patients with ASS and patients with "other" diagnoses. Convulsions and mental impairment were reported in patients with OTC and CPS. These observations are consistent with literature reports that patients with enzyme deficiencies occurring earlier in the urea cycle (i.e., OTC and CPS) tend to be more severely affected.

Adverse event profiles did differ by age group. Patients ≤ 30 days of age had more blood and lymphatic system disorders and vascular disorders (specifically hypotension), while patients > 30 days of age had more gastrointestinal disorders (specifically nausea, vomiting and diarrhea).

Other Less Common Adverse Events Occurring in < 3% of Patients

Less common adverse events that could represent drug-induced reactions or are characterized as severe are listed below by body system.